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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT00881933
Other study ID # KSPHO-SCT0804
Secondary ID
Status Recruiting
Phase Phase 1/Phase 2
First received April 14, 2009
Last updated March 23, 2012
Start date November 2008
Est. completion date September 2012

Study information

Verified date March 2012
Source The Korean Society of Pediatric Hematology Oncology
Contact Hyoung Jin Kang, M.D, Ph.D
Phone 82 2 2072 3304
Email kanghj@snu.ac.kr
Is FDA regulated No
Health authority Korea: Food and Drug Administration
Study type Interventional

Clinical Trial Summary

Severe aplastic anemia is a fatal disease and patients without HLA matched siblings need alternative treatment option. Cord blood transplantation (CBT) has become an alternative treatment means in various diseases, but it has not been proved to be good for severe aplastic anemia. Double units CBT is proposed to have better engraftment potential and and we reported successful double units UCBT after engraftment failure with single unit with promising result. To increase the engraftment potential, fludarabine, cyclophosphamide plus TBI conditioning regimen for double units cord blood transplantation was proposed for the patient with severe aplastic anemia without HLA-matched donor.


Recruitment information / eligibility

Status Recruiting
Enrollment 5
Est. completion date September 2012
Est. primary completion date September 2012
Accepts healthy volunteers No
Gender Both
Age group 1 Year to 21 Years
Eligibility Inclusion Criteria:

- Diagnosis of severe aplastic anemia defined by any two or three peripheral blood criteria and either marrow criterion.

- Peripheral blood

- Neutrophils < 0.5 x 109/l

- Platelets < 20 x 109/l

- Corrected reticulocytes < 1%

- Bone marrow

- Severe hypocellularity (< 25%)

- Moderate hypocellularity (25-30%) with hematopoietic cells representing < 30% of residual cells

- No prior hematopoietic stem cell transplantation.

- Age: no limits.

- Performance status: ECOG 0-2.

- Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.

- Heart: a shortening fraction > 30%, ejection fraction > 45%.

- Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal.

- Kidney: creatinine <2 × normal or a creatinine clearance (GFR) > 60 ml/min/1.73m2.

- Patients must lack any active viral infections or active fungal infection.

- No appropriate donor

- Appropriate cord blood is available: matched at least in 4/6 of A, B, DR loci.

- Patients (or one of parents if patients age < 19) should sign informed consent.

Exclusion Criteria:

- Pregnant or nursing women.

- Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.

- Psychiatric disorder that would preclude compliance.

- Congenital aplastic anemia including Fanconi anemia.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

Other:
Cyclophosphosphamide, TBI
fludarabine (30 mg/m2 once daily i.v. on days -7, -6, -5, -4, -3, & -2) (if body weight < 10 kg or if age < 1 year: 1.33 mg/kg) cyclophosphamide (60 mg/kg once daily i.v. on days -9, & -8) total body irradiation (3G on days -1)

Locations

Country Name City State
Korea, Republic of Seoul National University Hospital Seoul

Sponsors (1)

Lead Sponsor Collaborator
The Korean Society of Pediatric Hematology Oncology

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type Measure Description Time frame Safety issue
Primary To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for cord blood transplantation (CBT) in severe aplastic anemia. From Oct 2008. to Sep 2011 No
Secondary To evaluate the incidence and severity of toxicity and treatment related mortality From Oct 2008. to Sep 2011 No
Secondary To evaluate overall and event free survival rate From Oct 2008. to Sep 2011 No
Secondary To evaluate acute and chronic GVHD From Oct 2008. to Sep 2011 No
Secondary To evaluate immunologic recovery after CBT From Oct 2008. to Sep 2011 No
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